NEW YORK, N.Y. – April 30, 2015 – CereSpir™ Incorporated, a company developing CSP-1103, a first-in-class modulator of the brain’s innate immune system, as a potential treatment for Alzheimer’s disease (AD), announced today it has appointed Sophie Egholm as Chief Operating Officer. Ms. Egholm, a medicinal chemist and pharmacologist by training, has spent her entire career in the pharmaceutical industry holding increasingly senior management positions based on her excellent track record in Business, Finance and Operations Management, with significant experience in clinical trials for Alzheimer’s disease and related therapeutic areas.
CereSpir™ is developing CSP-1103, an orally delivered tablet, as a potential first disease-modifying treatment for Mild Cognitive Impairment due to AD targeting the stage of symptomatic disease before the onset of dementia. CSP-1103 has undergone Phase 2 testing in people with MCI, and CereSpir is finalizing the Phase 3 program based on input from the U.S. Food Drug Administration, European Medicines Agency and Japanese Pharmaceuticals and Medical Device Agency (PMDA). The Company is planning a parallel orphan drug development program in Batten disease.
Ms. Egholm’s industry career includes as Head of Operations and President of the Clinical Trials Business Unit at Cogstate Ltd, a cognitive science and rater training company, and as Associate Director, Strategic Alliances, Worldwide Business Development at Pfizer. She was also Global Marketing Manager at UCB BIOPRODUCTS in Belgium. Egholm earned her Master’s degree in Engineering from the Free University of Brussels and an Advanced Master’s in Medical Chemistry and Pharmacology from Lille Pharmacy University in Belgium.
“I am delighted to welcome Sophie as Chief Operating Officer, a role for which she is superbly qualified. Her considerable breadth of experience and extraordinary talents will be invaluable to help grow the company and achieve our business objectives,” said Daniel G. Chain, PhD, CereSpir’s President and Chief Executive Officer.
“I am excited to join CereSpir and very pleased to have this opportunity to help the company succeed in its critically important mission that could benefit of millions of people affected by Alzheimer’s disease and other neurodegenerative conditions,” said Sophie Egholm.
CSP-1103 is a novel, first-in-class, orally administered modulator of the brain’s innate immune system that helps clear the brain of toxic protein aggregates and reduce production of harmful cytokines. The Phase 2 clinical program generated preliminary safety and efficacy data in approximately 100 people half of whom received CSP-1103 for up to 2 years. Data from this study were presented at the Alzheimer’s Association International Conference in 2013 and 2014. CSP-1103 has been shown to inhibit brain amyloid beta (Aβ) plaque deposition, reduce tau pathology and neuro-inflammation and reverse associated memory deficits in AD transgenic mouse models. These changes appear to be associated with the observation that CSP-1103 can restore normal microglial function by increasing phagocytosis and decreasing production of pro-inflammatory cytokines. Chronic dysfunction of microglia is increasingly believed to play an important role at the very beginnings of AD with several newly discovered genetic risk factors supporting this idea. These effects of CSP-1103 may translate into preventing the memory loss that is the hallmark of AD. Microglia form an important part of the innate immune system of the brain and are small cells that migrate through the brain to remove waste products, such as the amyloid aggregates that cause inflammation and irreversible damage to nerve cells. We believe that the numerous beneficial pharmacological properties, which have been observed for CSP-1103, are mediated via its binding to a single recently identified molecular target – the intracellular domain of the amyloid precursor protein (AICD) which is hypothesized to mediate pro-inflammatory properties of microglia. CSP-1103 is the only small molecule known to bind to AICD and to interfere with the expression of target genes involved in neuroinflammation.
The results from previous human clinical studies are consistent with a large body of preclinical data. Thus, for example, short term treatment of patients with MCI was shown to be associated with lower levels of cerebrospinal fluid (CSF) inflammatory markers (sCD40L and TNF-α) in a dose-related fashion while prolonged treatment is associated with lower levels of a CSF marker of neurodegeneration (total tau). Also, long-term treatment suggested possible cognitive benefit that correlated with the lower levels of tau providing a link between mechanism of action and clinical benefit.
About CereSpir™ Incorporated
CereSpir is a private pharmaceutical company in New York, New York, U.S.A., founded by Daniel G. Chain, Ph.D. The Company assumed full rights to CSP-1103 in November 2013 and became the official sponsor of the IND in March 2014. CSP-1103 is a novel, first-in-class, small molecule modulator of the brain’s innate immune system that has utility for the treatment of AD and other neurodegenerative diseases. In a Phase 2 trial in MCI patients, CSP-1103 was both well tolerated and appeared to both reduce brain inflammation and result in sustained cognition. MCI causes a slight but noticeable and measurable decline in cognitive abilities, including memory and thinking skills. Most people with MCI progress to an Alzheimer’s disease (AD) diagnosis which currently affects more than 5 million Americans, 7 million Europeans and, in total, about 44 million people worldwide according to the most recent report by the Alzheimer’s Association. Current treatments for AD are symptomatic and temporary. There are no drugs approved which slow or halt the mental decline associated with this terrible disease. CereSpir is leading the way with a completely novel approach to AD by reducing inflammation and increasing clearance of harmful protein aggregates in the brain. This novel approach targets aggregates of both beta amyloid (Aβ) and Tau protein that together cause irreversible damage to nerve cells and are the characteristic pathology of AD. CereSpir is planning to conduct a Phase 3 program for CSP-1103 using an enrichment strategy of biomarkers and other diagnostic criteria that would limit enrollment to patients with MCI due to AD. The company also plans a parallel orphan drug development program in Batten disease (Juvenile Neuronal Ceroid Lipofuscinosis), in which microglial impairment appears to be a major driver of neuronal loss.